Ver­sant care­ful­ly sets the stage for a $68M de­but of a biotech play­er fo­cused on syn­thet­ic lethal­i­ty

Over the past 18 months, the grow­ing team at Re­pare Ther­a­peu­tics has been qui­et­ly set­ting up shop in Ver­sant Ven­tures’ dis­cov­ery ops and fo­cus­ing on syn­thet­ic lethal­i­ty — the ther­a­peu­tic in­ter­play be­tween ge­net­ic mu­ta­tions and can­cer. Us­ing CRISPR gene edit­ing tech as an in­ter­ro­ga­tion tool, they’ve been play­ing with well known tar­gets like p53 and BR­CA1 to find out how they con­spire with oth­er mu­ta­tions in the de­struc­tion of a ma­lig­nan­cy, fol­low­ing the trail of le­sions in the pur­suit of new ther­a­pies that can work in the same fun­da­men­tal way those PARP in­hibitors you’ve been hear­ing so much about can fight can­cer.

The goal was to mas­ter syn­thet­ic lethal­i­ty and iden­ti­fy the first cou­ple of tar­gets to go af­ter in the clin­ic. They lined up their team, now at 20, li­censed in dis­cov­ery work from NYU on a poly­merase im­pli­cat­ed in nu­mer­ous can­cers, and to­day will come out of stealth mode with a $68 mil­lion round and at least a four-year run­way with plans to start tack­ling clin­i­cal work in late 2019.

Their first pro­gram in­volves DNA-di­rect­ed DNA poly­merase theta — PolQ — in­clud­ed in a path­way that re­pairs dou­ble-strand breaks in can­cer cells. And while they have a ways to go be­fore they be­gin hu­man stud­ies, the in­vestors are send­ing a mes­sage that the amount of cash they’re bet­ting on Re­pare in­di­cates a high lev­el of con­fi­dence for their longterm suc­cess.

Jer­el Davis

“In terms of the in­cu­ba­tion pe­ri­od,” says Jer­el Davis, man­ag­ing di­rec­tor at Ver­sant, “this com­pa­ny is de­cep­tive­ly ma­ture.”

“The time we’ve been able to spend in stealth al­lowed us to build the com­pa­ny out of the spot­light,” says CEO Lloyd Se­gal. In Mon­tre­al they have de­vel­oped their med­i­c­i­nal chem­istry team un­der Cameron Black, the for­mer head of chem­istry at Mer­ck Frosst who joined a lit­tle more than a year ago. And when Ver­tex re­cent­ly shut down in Mon­tre­al, they swooped in, grab­bing an ex­pe­ri­enced team with a long track record of work­ing to­geth­er.

Lloyd Se­gal

With­in hours of the shut­down, they had 13 new re­cruits out of 14 of­fers.

R&D head Michael Zin­da, who led As­traZeneca On­col­o­gy iMed Bio­science group in Boston, is head­ing up the oth­er group in Cam­bridge, MA.

Ver­sant has gath­ered an im­pres­sive group of mar­quee in­vestors to back their up­start. MPM Cap­i­tal came in along­side as the lead in­vestor on the Se­ries A, with Cel­gene’s Swiss of­fice tak­ing part along with FTQ and BDC Ven­tures.

“We could have tak­en more mon­ey,” Se­gal tells me. “We had more than that on the ta­ble. But we be­lieved that was the kind of mon­ey that we hope will take two com­pounds in­to the clin­ic in late ’19 or ear­ly 2020.”

Ver­sant had a front row seat on some of the ear­ly work on syn­thet­ic lethal­i­ty at Clo­vis while it was work­ing on PARP, says Davis. “This is a field we know and our con­vic­tion is that PARP is the tip off the ice­berg.”

Re­pare has the plat­form that they plan to use to map out some of the rest of the un­ex­plored ter­ri­to­ry.

Even with­out R&D part­ners Se­gal feels that he has enough cash to make their way through at least the first 4 years. Add in the prospect of some ma­jor league play­ers com­ing in to part­ner on key prospects, and that run­way will stretch fur­ther. If it all plays out ac­cord­ing to plan, Se­gal adds, this will be their last ven­ture raise.

It’s an am­bi­tious plan, with some stel­lar sci­en­tif­ic ad­vis­ers ready to leaned guid­ance. The full sci­en­tif­ic ad­vi­so­ry board in­cludes:

– Samuel Apari­cio, pro­fes­sor in the de­part­ment of pathol­o­gy and lab­o­ra­to­ry med­i­cine at the Uni­ver­si­ty of British Co­lum­bia.

– Jim Carmichael, head of the pro­tein home­osta­sis the­mat­ic cen­ter of ex­cel­lence at Cel­gene. He pre­vi­ous­ly was UK re­gion­al di­rec­tor of med­ical sci­ence at As­traZeneca fol­low­ing its ac­qui­si­tion of Ku­DOS, where he was CMO and re­spon­si­ble for clin­i­cal de­vel­op­ment of ola­parib.

– Ron­ny Drap­kin, di­rec­tor of the Penn Ovar­i­an Can­cer Re­search Cen­ter and di­rec­tor of gy­ne­co­log­ic can­cer re­search at the Uni­ver­si­ty of Penn­syl­va­nia.

– Lau­rie Glim­ch­er, pres­i­dent and CEO of the Dana-Far­ber Can­cer In­sti­tute.

– Mark Pe­gram, di­rec­tor of the breast can­cer on­col­o­gy pro­gram at Stan­ford Women’s Can­cer Cen­ter and co-di­rec­tor of Stan­ford’s mol­e­c­u­lar ther­a­peu­tics pro­gram.

– Richard Wood, pro­fes­sor of mol­e­c­u­lar bi­ol­o­gy at the Uni­ver­si­ty of Texas MD An­der­son Can­cer Cen­ter.

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others.

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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George Yancopoulos (Regeneron)

UP­DAT­ED: Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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Look­ing for 'ex­ter­nal in­no­va­tion,' Boehringer In­gel­heim re­serves $500M+ for new Shang­hai hub

Now that Boehringer Ingelheim’s bet on contract manufacturing in China has paid off, the German drugmaker is anteing up more to get into the research game.

Boehringer has set aside $507.9 million (€451 million) for a new External Innovation Hub to be built in Shanghai over five years. The site will become one of its “strategic pillars” as the team strives to get 71 approvals — either for new products or indications — by 2030, said Felix Gutsche, president and CEO of Boehringer Ingelheim China.

Vas Narasimhan, Novartis CEO (Patrick Straub/​EPA-EFE/​Shutterstock)

No­var­tis pays $678M for kick­back scheme as Vas Narasimhan tries to dis­tance phar­ma gi­ant from shady be­hav­ior

Novartis has reached another large settlement to resolve misconduct allegations, agreeing to pay more than $678 million to settle claims that it had spent hundreds of millions of dollars on lavish dinners, so-called speaking fees and expensive alcohol “that were nothing more than bribes” to get doctors to prescribe Novartis medications.

The top-shelf alcohol and lavish meals included a $3,250 per person night at Nobu in Dallas, a $672-per person dinner at Washington DC’s Smith & Wollensky and a $314 per person meal at Sushi Roku in Pasadena, according to the Justice Department complaint. There were at least 7 trips to Hooters and fishing trips in Alaska and off the Florida coast. Each of these events were supposed to be “speaker programs” where doctors educated other doctors on a drug, but the DOJ alleged many were “bogus” wine-and-dine events where the drug was barely mentioned, if at all.  (“Nobody presented slides on the fishing trips,” the complaint says.)

No­vavax snags Ben Machielse for CMC and pro­motes a trio of staffers; Mar­ty Du­vall lands an­oth­er CEO post at On­copep­tides

Novavax has been making waves recently by securing a $384 million commitment from CEPI to cover R&D and manufacturing for its Covid-19 vaccine while also spending $167 million on a 150,000 square-foot facility. The Maryland biotech continues to shore up its leadership team as well, bringing in Ben Machielse as their EVP of CMC just a couple weeks after nabbing AstraZeneca vet Filip Dubrovsky as their new CMO. Machielse was president and CEO of Vtesse from 2014-17, and before that, he also spent more than 11 years at MedImmune and was EVP of operations for the back half of his tenure.

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Dan Gold, MEI Pharma CEO

De­vel­op­ment part­ners at MEI, Helsinn dump a high-risk PhI­II AML study af­ter con­clud­ing it would fail sur­vival goal

Four years after Switzerland’s Helsinn put $25 million of cash on the table for an upfront and near-term milestone to take MEI Pharma’s drug pracinostat into a long-running Phase III trial for acute myeloid leukemia, the partners are walking away from a clinical pileup.

The drug — an HDAC inhibitor — failed to pass muster during a futility analysis, as researchers concluded that pracinostat combined with azacitidine wasn’t going to outperform the control group in the pivotal.

No­var­tis los­es biosim­i­lar ap­peal as court up­holds a 31-year mo­nop­oly by Am­gen's En­brel

A new court ruling has strengthened Amgen’s grip on the IP estate around Enbrel, keeping biosimilars of the autoimmune and inflammatory drug at bay until 2029.

Novartis, the patent challenger, isn’t throwing in the towel yet. In a statement noting the failed appeal, its generics division Sandoz noted its reviewing options, “including potential appeal to US Supreme Court.”

It’s been almost four years since the FDA approved Erelzi, Sandoz’s copycat version of Enbrel. While sales of the Pfizer-partnered drug in the US — the market Amgen is in charge of — have dipped slightly during that time, it remains a solid megablockbuster with 2019 revenue slightly above $5 billion.